Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.

Shabanpoor, Fazel

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy. [electronic resource] - Nucleic acid therapeutics Jun 2017 - 130-143 p. digital

Publication Type: Journal Article

2159-3345

10.1089/nat.2016.0652 doi


Animals
Animals, Newborn
Apolipoproteins E--chemical synthesis
Biomarkers--blood
Blood-Brain Barrier--chemistry
Brain--cytology
Cell Survival--drug effects
Disease Models, Animal
Exons
Fibroblasts--drug effects
Hepatocytes--drug effects
Humans
Kidney--chemistry
Mice
Morpholinos--chemistry
Muscular Atrophy, Spinal--drug therapy
Nanoconjugates--analysis
Peptides--chemical synthesis
Phenotype
Quadriceps Muscle--chemistry
Survival of Motor Neuron 2 Protein--drug effects